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D. Pangonis
D. Pangonis
GM2 gangliosidoses
Clinical endpoint
Sandhoff disease
Quality of life
Ataxia-telangiectasia
1
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5
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A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia.
2021
Trials
T. Fields
Marc C. Patterson
T Bremova-Ertl
G. Belcher
I. Billington
Grant C. Churchill
W. Davis
W. Evans
S. Flint
Antony Galione
U Granzer
J Greenfield
R. Karl
R. Kay
D. Lewi
T. Mathieson
T. Meyer
D. Pangonis
Frances M. Platt
L. Tsang
C. Verburg
M. Factor
Michael Strupp
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Citations (5)
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